Evidence map›Paper›PMID 38372904›Full record

Trial reportJournal of neuro-oncology2024

Imaging as an early biomarker to predict sensitivity to everolimus for progressive NF2-related vestibular schwannoma.

Phioanh Leia Nghiemphu, Jeremie Vitte, Eva Dombi, Thien Nguyen, Naveed Wagle, Akira Ishiyama, Ali R Sepahdari, David Cachia, Brigitte C Widemann, Derald E Brackmann and 3 more

Registry-linked trialOpen access · hybridAbstract readClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of neuro-oncology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01345136 (A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma), which is not on this map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
1.8field-weighted citation impact, top 15% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT01345136 phase2terminatednot on this map

A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma

TypeinterventionalSponsorJonsson Comprehensive Cancer CenterRan2015 to 2024Enrolled4ConditionsNeurofibromatosis Type 2, Neuroma, AcousticArmsRAD001, everolimus
3 · Its place in the literature

Who cites it

6 citing papers in PubMed, 5 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors at 6 institutions in 2 countries.

Phioanh Leia NghiemphuDepartment of Neurology, UCLA Neuro‑Oncology Program, David Geffen School of Medicine and Jonsson Comprehensive Cancer Center (JCCC), University of California, Los Angeles, Los Angeles, CA, USA.ORCID http://orcid.org/0000-0003-4064-6079
Jeremie VitteDepartment of Head and Neck Surgery, David Geffen School of Medicine and Jonsson Comprehensive Cancer Center (JCCC), University of California, Los Angeles, 675 Charles E Young Dr. S, MRL 2240, Los Angeles, CA, 90095-7286, USA.ORCID http://orcid.org/0000-0002-2567-7988
Eva DombiPediatric Oncology Branch, National Cancer Institute, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-5323-6995
Thien NguyenDepartment of Neurology, UCLA Neuro‑Oncology Program, David Geffen School of Medicine and Jonsson Comprehensive Cancer Center (JCCC), University of California, Los Angeles, Los Angeles, CA, USA.ORCID http://orcid.org/0000-0003-2348-0556
Naveed WagleDepartment of Medicine, Division of Medical Oncology, Norris Cancer Center, University of Southern California, Los Angeles, CA, USA.ORCID http://orcid.org/0009-0007-8072-0348
Akira IshiyamaDepartment of Head and Neck Surgery, David Geffen School of Medicine and Jonsson Comprehensive Cancer Center (JCCC), University of California, Los Angeles, 675 Charles E Young Dr. S, MRL 2240, Los Angeles, CA, 90095-7286, USA.ORCID http://orcid.org/0000-0002-1913-0812
Ali R SepahdariDepartment of Radiology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.ORCID http://orcid.org/0000-0002-3806-1728
David CachiaDepartment of Neurosurgery, Division of Neuro-oncology, Medical University of South Carolina, Charleston, SC, USA.ORCID http://orcid.org/0000-0002-5860-8452
Brigitte C WidemannPediatric Oncology Branch, National Cancer Institute, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-9198-7175
Derald E BrackmannDepartment of Otolaryngology and Neurotology, House Clinic and Research Institute, Los Angeles, CA, USA.
Joni K DohertyCenter for Neural Tumor Research, House Research Institute, Los Angeles, CA, USA.ORCID http://orcid.org/0000-0002-3846-3954
Michel KalamaridesDepartment of Neurosurgery, Hôpital Pitié-Salpêtrière, APHP, Sorbonne Université, Paris, France.ORCID http://orcid.org/0000-0003-1037-9707
Marco GiovanniniDepartment of Head and Neck Surgery, David Geffen School of Medicine and Jonsson Comprehensive Cancer Center (JCCC), University of California, Los Angeles, 675 Charles E Young Dr. S, MRL 2240, Los Angeles, CA, 90095-7286, USA. mgiovannini@mednet.ucla.edu.ORCID http://orcid.org/0000-0001-5183-8506
University of California, Los Angeles · USNational Cancer Institute · USUniversity of Southern California · USHouse Clinic · USMedical University of South Carolina · USSorbonne Université · FR

Funding

UCLA SPORE in Brain CancerP50CA211015 · NCI · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI David A. Nathanson · 2017 to 2026
$25.2M
Therapies for patients with rare tumors and genetic tumor predispositionZIASC010354 · NCI · DIVISION OF CLINICAL SCIENCES - NCI · PI WIDEMANN, BRIGITTE · 2009 to 2025
$19.2M
Congressionally Directed Medical Research Programs W81XWH-12-1-0116NCI NIH HHS P50 CA211015
6 · The paper itself

Abstract

purposeNF2-related schwannomatosis (NF2) is characterized by bilateral vestibular schwannomas (VS) often causing hearing and neurologic deficits, with currently no FDA-approved drug treatment. Pre-clinical studies highlighted the potential of mTORC1 inhibition in delaying schwannoma progression. We conducted a prospective open-label, phase II study of everolimus for progressive VS in NF2 patients and investigated imaging as a potential biomarker predicting effects on growth trajectory.

methodsThe trial enrolled 12 NF2 patients with progressive VS. Participants received oral everolimus daily for 52 weeks. Brain imaging was obtained quarterly. As primary endpoint, radiographic response (RR) was defined as ≥ 20% decrease in target VS volume. Secondary endpoints included other tumors RR, hearing outcomes, drug safety and quality of life (QOL).

resultsEight participants completed the trial and four discontinued the drug early due to significant volumetric VS progression. After 52 weeks of treatment, the median annual VS growth rate decreased from 77.2% at baseline to 29.4%. There was no VS RR and 3 of 8 (37.5%) participants had stable disease. Decreased or unchanged VS volume after 3 months of treatment was predictive of stabilization at 12 months. Seven of eight participants had stable hearing during treatment except one with a decline in word recognition score. Ten of twelve participants reported only minimal changes to their QOL scores.

conclusionsVolumetric imaging at 3 months can serve as an early biomarker to predict long-term sensitivity to everolimus treatment. Everolimus may represent a safe treatment option to decrease the growth of NF2-related VS in patients who have stable hearing and neurological condition. TRN: NCT01345136 (April 29, 2011).

Indexed as

Neurofibromatosis 2Neuroma, AcousticBiomarkersEverolimusHumansQuality of LifeTreatment OutcomeBiomarkersEverolimusClinical trialEverolimusMammalian target of rapamycin (mTOR) inhibitorsNF2Quantitative imaging biomarkersVestibular schwannoma

Identifiers

PMID38372904
PMCPMC11023969
OpenAlexW4391931000

What OpenQuestion holds

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LicenceCC BY
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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.